BUFFALO, NY- February 7, 2024 – A new research paper was published in Aging Volume 16, Issue 2, entitled, “PROX1 interaction with α-SMA-rich cancer-associated fibroblasts facilitates colorectal cancer progression and correlates with poor clinical outcomes and therapeutic resistance.” The tumor microenvironment (TME) plays a vital role in tumor progression through...
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TAMPA, Fla. — Psilera, a leading biopharmaceutical company focused on developing novel treatments for neurological disorders, is pleased to announce the selection of behavioral variant frontotemporal dementia (bvFTD) as the lead indication for its groundbreaking drug candidate, PSIL-006. This devastating form of early-onset dementia currently lacks approved treatments to manage symptoms...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that both the United States Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for PTC923 for the treatment of patients with hyperphenylalaninemia. Phenylketonuria (PKU) accounts for 98% of all hyperphenylalaninemia cases and is a metabolic condition that...
WARREN, N.J. — PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. “The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe,” said Matthew B....
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil. FCS is a rare genetic disease which results in significant disease burden to patients including potentially fatal pancreatitis...
SOUTH PLAINFIELD, N.J. – PTC Therapeutics, Inc. (NASDAQ: PTCT) today introduced a new no-charge, patient-initiated testing program in the United States for individuals who have symptoms consistent with, or a diagnosis of, cerebral palsy (CP) with no evidence of brain injury. CP is a group of disorders that affect a person’s ability to...
SOUTH PLAINFIELD, N.J. — PTC Therapeutics, Inc. (PTC) today announced the receipt of two government grants to support clinical development programs in rare disorders. The U.S. Department of Defense Neurofibromatosis Research Program has awarded an $822,345 grant to support a new open-label Phase 2 clinical trial of PTC’s product candidate...
Washington, DC – The FDA will host Rare Disease Day, a virtual public meeting, on March 1, 2024 in global observance of Rare Disease Week. This year’s Rare Disease Day is dedicated to patients and health care professionals. Panels will discuss: The legal framework for approving studies and medical products...
LACHEN, Switzerland — Octapharma announced today the publication of the results from the clinical study WIL-31, which investigated the efficacy and safety of prophylaxis with wilate®, a von Willebrand factor/factor VIII (VWF/FVIII) concentrate, in patients with all types of von Willebrand disease (VWD). The results have been published in the...
WASHINGTON — Think your job’s tedious? Try beheading 100 mosquitoes an hour. Gently, no smushing allowed. Malaria parasites lurk in these mosquitoes’ salivary glands, and a small company on the outskirts of the nation’s capital needs them unharmed for a dramatic test — attempting the first live vaccine to fight...